Inherited Metabolic Disorders (IMD)IMD-002NCT04008849
A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456
An observational study of Inherited Metabolic Disorders (IMD), run by Magenta Therapeutics, Inc. at 1 site in United States.
Trial summary
A follow-up study to evaluate the safety and clinical outcomes of patients with inherited metabolic disorders (IMD) who have undergone hematopoietic stem cell transplantation (HSCT) with MGTA-456 Read more about this study
- Who is it for?
- Ages Up to 16 years
- Conditions the study is for
- Inherited Metabolic Disorders (IMD)
- What is being tested?
- Safety and efficacy assessments
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 21 May 2019to 8 Oct 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 16 years
- All sexes
- No healthy volunteers
- Child
Study population: Subjects with an inherited metabolic disorder (IMD) that were treated with MGTA-456
Who may be able to join
- An Institutional Review Board (IRB)/Independent Ethics Committee-approved written informed consent form must be signed and dated by the patient or legal guardian. Study assents will also be prepared for children and adolescents to review when applicable.
- Patient completed an IMD study in the MGTA-456 program and was administered MGTA-456 for HSCT.
Who cannot join
- •Patients enrolled in an IMD study in the MGTA-456 program who did not receive MGTA-456 or were withdrawn from the core study.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota
Minneapolis, Minnesota, 55455
About this study
Official title: A Follow-Up Study to Evaluate the Safety and Clinical Outcomes of Patients With Non-Malignant Disease Who Have Undergone Hematopoietic Stem Cell Transplantation With MGTA-456
A follow-up study to evaluate the safety and clinical outcomes of patients with inherited metabolic disorders (IMD) who have undergone hematopoietic stem cell transplantation (HSCT) with MGTA-456
Read the detailed description
This is a follow-up study to evaluate the long-term safety and efficacy outcomes of patients with inherited metabolic disorders (IMDs) who received MGTA-456 for HSCT in the core study. MGTA-456 is an expanded CD34+ cell therapy product candidate given after myeloablative conditioning to induce rapid and sustained hematopoietic engraftment. In patients with selected IMDs, transplant is expected to replace defective or missing protein, and preserve neurodevelopment. Patients with Hurler syndrome (also referred to as mucopolysaccharidosis-1H (MPS-1H)), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD) enrolled in the core study will be eligible to participate in this follow-up evaluation.
- Conditions
- Inherited Metabolic Disorders (IMD)
- Being tested
- Safety and efficacy assessments
- Keywords
- Inherited Metabolic Disorders, Hematopoietic Stem Cell Transplant, MGTA-456, Umbilical Cord Blood Transplant, Hurler Syndrome, Cerebral Adrenoleukodystrophy, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy
- Medical subject headings
- Mucopolysaccharidosis I, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Safety and efficacy assessmentsOther
Long term safety and clinical outcomes
| Study group | Role | What they receive |
|---|---|---|
| Subjects treated with MGTA-456 | MGTA-456 is an investigational expanded CD34+ cell therapy Receives: Other: Safety and efficacy assessments |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of related adverse events
Measured: 2 years
Incidence of serious adverse events
Measured: 2 years
Incidence of late hematological graft failure
Measured: 2 years
Incidence of chronic graft versus host disease
Measured: 2 years
Overall survival
Measured: 2 years
Event-free survival
Measured: 2 years
Change in cALD Neurologic Function Score over time
Measured: 2 years
Proportion of subjects without gadolinium enhancement on MRI over time
Measured: 2 years
Alpha-iduronidase blood enzyme level (nmol/hr/mg) in Hurler patients
Measured: 2 years
Very long chain fatty acid blood level (ug/mL) in cALD patients
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2019
- Study start21 May 2019
- Primary completion8 Oct 2020
- Study completion8 Oct 2020
Why it stopped: Due to low enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Inherited Metabolic Disorders (IMD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Magenta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Paul J Orchard, MD, Principal investigator, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04008849
- Sponsor's protocol ID
- IMD-002
- Acronym
- IMD-002
- Status last verified
- Mar 2021
- First submitted
- 23 May 2019
- Last updated
- 15 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Follow-Up Study to Evaluate the Safety and Clinical Outcomes of Patients With Non-Malignant Disease Who Have Undergone Hematopoietic Stem Cell Transplantation With MGTA-456
- ClinicalTrials.gov ID
- NCT04008849
- Acronym / sponsor ID
- IMD-002 / IMD-002
- Conditions
- Inherited Metabolic Disorders (IMD)
- Keywords
- Inherited Metabolic Disorders, Hematopoietic Stem Cell Transplant, MGTA-456, Umbilical Cord Blood Transplant, Hurler Syndrome, Cerebral Adrenoleukodystrophy, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Due to low enrollment
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Magenta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 21 May 2019 (actual)
- Primary completion
- 8 Oct 2020 (actual)
- Study completion
- 8 Oct 2020 (actual)
- First posted
- 5 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A follow-up study to evaluate the safety and clinical outcomes of patients with inherited metabolic disorders (IMD) who have undergone hematopoietic stem cell transplantation (HSCT) with MGTA-456
Detailed description
This is a follow-up study to evaluate the long-term safety and efficacy outcomes of patients with inherited metabolic disorders (IMDs) who received MGTA-456 for HSCT in the core study. MGTA-456 is an expanded CD34+ cell therapy product candidate given after myeloablative conditioning to induce rapid and sustained hematopoietic engraftment. In patients with selected IMDs, transplant is expected to replace defective or missing protein, and preserve neurodevelopment. Patients with Hurler syndrome (also referred to as mucopolysaccharidosis-1H (MPS-1H)), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD) enrolled in the core study will be eligible to participate in this follow-up evaluation.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Subjects treated with MGTA-456 | – | Other: Safety and efficacy assessmentsMGTA-456 is an investigational expanded CD34+ cell therapy |
Who may qualify
- Ages
- Up to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Subjects with an inherited metabolic disorder (IMD) that were treated with MGTA-456
Key inclusion criteria (2 listed)
- An Institutional Review Board (IRB)/Independent Ethics Committee-approved written informed consent form must be signed and dated by the patient or legal guardian. Study assents will also be prepared for children and adolescents to review when applicable.
- Patient completed an IMD study in the MGTA-456 program and was administered MGTA-456 for HSCT.
Key exclusion criteria (1 listed)
- •Patients enrolled in an IMD study in the MGTA-456 program who did not receive MGTA-456 or were withdrawn from the core study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Safety and efficacy assessmentsLong term safety and clinical outcomes | Other | Subjects treated with MGTA-456 |
Primary outcome
Incidence of related adverse events
Time frame: 2 years
Incidence of serious adverse events
Time frame: 2 years
Incidence of late hematological graft failure
Time frame: 2 years
Incidence of chronic graft versus host disease
Time frame: 2 years
Overall survival
Time frame: 2 years
Event-free survival
Time frame: 2 years
Change in cALD Neurologic Function Score over time
Time frame: 2 years
Proportion of subjects without gadolinium enhancement on MRI over time
Time frame: 2 years
Alpha-iduronidase blood enzyme level (nmol/hr/mg) in Hurler patients
Time frame: 2 years
Very long chain fatty acid blood level (ug/mL) in cALD patients
Time frame: 2 years
Results and safety
The study was stopped early (reason given: Due to low enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Mar 2021.
- Individual participant dataSharing planned: No
Related trials for Inherited Metabolic Disorders (IMD)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Paul J Orchard, MD principal investigator
University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Inherited Metabolic Disorders (IMD), run by Magenta Therapeutics, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 15 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Due to low enrollment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of related adverse events (time frame: 2 years). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Mar 2021.